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SMA Europe 2024

Coming soon
08:00 AM
Duration 90mins Ghent, Belgium
Correlation of individual SMN protein with observed efficacy in the risdiplam▼ clinical trials
Y Cleary, P Grimsey, CA Chiriboga, E Mercuri, T McIver, R Thomas, RS Scalco, H Kletzl
Duration 90mins Ghent, Belgium
Long-term comparative efficacy and safety of risdiplam▼ versus nusinersen in children with Type 1 SMA
CS Sutherland, N Hawkins, G Sajeev, R Evans, A Mahajan, DA Scott, J Nam, E Gaki, C, Kokaliaris
Duration 90mins Ghent, Belgium
FIREFISH Parts 1 and 2: 48-month safety and efficacy of risdiplam▼ in Type 1 SMA
R Masson, M Mazurkiewicz-Beldzinska, G Baranello, O Boespflug-Tanguy, JW Day, N Deconinck, A Klein, E Mercuri, K Rose, L Servais, D Vlodavets, H Xiong, E Zanoteli, M El-Khairi, M Gerber, K Gorni, H Kletzl, L Palfreeman, A Dodman, BT Darras
Duration 90mins Ghent, Belgium
MANATEE: GYM329 (RO7204239) in combination with risdiplam▼ treatment with SMA
L Servais, BT Darras, J Morrow, F Muntoni,T Duong, M Rabbia, M Gerber, H Kletzl, E Gaki, S Fletcher, R Scalco, E Mercuri
Duration 90mins Ghent, Belgium
Bioequivalence and food effect assessment for a room-temperature stable risdiplam▼ tablet formulation in healthy volunteers
H Kletzl, K Heinig, B Jaber, W Y Yeung, H Coleman, B Lomeli, L Buckley, A Varghese
Duration 90mins Ghent, Belgium
Protocol for a Delphi consensus panel on assessing clinically meaningful treatment outcomes in adults living with SMA
K Sully, CA Chiriboga, T Duong, Y Erbas, J Glascock, N Gusset, R Muni-Lofra, C O’Connell, JF Vazquez-Costa, MC Walter, CJ Guittari, J Cochrane, LD Townson, J Kufakwaro, A Marciniak, D Riley, J Nam
Duration 90mins Ghent, Belgium
Assessing the feasibility of TREAT-NMD registries for characterizing adult patients with SMA receiving risdiplam▼ treatment
A Simpson, T Carmona, M Batech, SY Lee, N Bennett, S Edwards, CS Sutherland, C Marini-Bettolo, S Thiele, M Jagut, J Haberlová, V Hodgkinson, R Forbes, LB Murphy, V Matyushenko
Duration 90mins Ghent, Belgium
A retrospective, non-interventional cohort study to compare adults with SMA receiving risdiplam ▼with untreated patients: Study design
A Simpson, M Batech, A Carmona, SY Lee, A Poll, S Edwards, CJ Guittari, CS Sutherland, C Marini-Bettolo, MC Walter, M Jagut, J Haberlová, V Hodgkinson, E Yiu, LB Murphy
Duration 90mins Ghent, Belgium
Real-World Outcomes of Infants who Initiate Risdiplam▼ Under 2 Months of Age for the Treatment of Spinal Muscular Atrophy (SMA) in the US
​​​Travis Dickendesher, Selina Tam, Dalia Moawad, S Edwards, Alex Simpson, Sheila Shapouri
01:30 PM
Duration 15mins Ghent, Belgium
RAINBOWFISH: Primary efficacy and safety data in risdiplam▼-treated infants with presymptomatic SMA
M A Farrar, R S Finkel, L Servais, D Vlodavets, E Zanoteli, M Al-Muhaizea, A Prufer, L Nelson,C Fischer, M Gerber, K Gorni, H Kletzl, L Palfreeman, E Gaki, P Fontoura, E Bertini
03:00 PM